Gene therapy could free blood disease patients from transfusions
Disease control
Not yet recruiting
This study tests a single dose of CS-101, a gene therapy, in 20 people aged 12 to 35 with β-thalassemia major, a severe blood disorder requiring regular transfusions. The goal is to see if it is safe and can help patients produce enough healthy hemoglobin to stop needing transfus…
Phase: PHASE2 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated May 12, 2026 13:41 UTC